The House of Representatives voted overwhelmingly on Wednesday to approve the 21st Century Cures Act, a sprawling bill to fund medical research and revamp how drugs and medical devices are approved by the Food and Drug Administration.
The bill passed on a vote of 392-26. The Senate could vote on the bill early next week. It has garnered support from a wide variety of advocacy groups, industry lobbyists and the Obama administration. One notable dissident is Sen. Elizabeth Warren, D-Mass., who said her colleagues have “let Big Pharma hijack the Cures bill.” […]
Advocates for biomedical research are happy to see billions of dollars in new funding for the NIH and associated projects. (The money will come, in part, from portions of the Affordable Care Act that are on the chopping block following the November election.)
The National Institutes of Health budget has slipped about 20 percent over the past decade in terms of buying power. The 21st Century Cures Act does little to offset that decline, but it does include billions of research dollars for some new high-profile projects, such as Vice President Biden’s push to accomplish 10 years of cancer research in half that time. […]
The bill also promises faster action on potential new drugs and devices. And this is where some of the concerns come in. […]
“There’s no doubt that patients would like to get treatments faster to the market, but they need to be assured those treatments are safe and effective for each of those marketed uses,” Carome says.
The new law would allow a drug approved for one purpose to be used for a related disease without going through the entire approval process, he said. Carome worries that measure could put patients at risk.
The Food and Drug Administration has been working faster on drug approvals, even without the new legislation. Drugs can be approved based on inferences that they work, but without proof of a long-term benefit. Researchers use a “surrogate endpoint,” such as tumor’s response to a drug, even though that response may not lead to a longer or healthier life.
The faster approach doesn’t always work out as planned. Diana Zuckerman, who heads the National Center for Health Research, points to a study that found 18 of the 36 cancer drugs recently approved on the basis of surrogate endpoints turned out not to help people live longer.
“What’s happening is we are flooding the market with medical products that don’t work very well, or we don’t know whether they work,” Zuckerman says.
She has a new study (slated for publication in JAMA Internal Medicine, but not yet in print) showing that often these new cancer drugs don’t even improve the quality of life for these cancer patients, despite the drugs’ soaring price tags.
Zuckerman say she’s concerned that the 21st Century Cures act will make it even easier for drug companies to use this express lane for approval. […]